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US 12,214,023 B2Granted
Compositions and methods for expressing factor IX
AssigneesIntellia Therapeutics, Inc., Regeneron Pharmaceuticals, Inc.
InventorsJonathan Douglas Finn, Hon-Ren Huang, Moitri Roy, KehDih Lai, Rachel Sattler, Christos Kyratsous, Cheng Wang
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Abstract
Compositions and methods for expressing Factor IX in a host cell or a population of host cells are provided. Also provided are engineered host cells expressing Factor IX.
Claims16
A method of expressing Factor IX in a cell or population of cells, comprising administering:
- i) a nucleic acid construct comprising a Factor IX protein coding sequence;
- ii) an RNA-guided DNA binding agent;
- and
- iii) a guide RNA (gRNA) comprising the sequence of SEQ ID NO:
- 8,
- thereby expressing Factor IX in a cell or population of cells.
3 more elements
A composition for use in expressing Factor IX in a cell, wherein the composition comprises:
- i) a nucleic acid construct comprising a Factor IX protein coding sequence;
- ii) an RNA-guided DNA binding agent;
- and
- iii) a guide RNA (gRNA) comprising the sequence of SEQ ID NO:
- 8.
2 more elements
A method of treating a Factor IX deficiency comprising administering to an individual with the Factor IX deficiency:
- i) a nucleic acid construct comprising a Factor IX protein coding sequence;
- ii) an RNA-guided DNA binding agent;
- and
- iii) a guide RNA (gRNA) comprising the sequence of SEQ ID NO:
- 8,
- thereby expressing the Factor IX protein in the individual.
3 more elements
Dependencies are inferred from the claim text.